Gene & Cell Therapy : Key Highlights in Vision, Heart, Neurological Treatments & More 🧬👁️❤️🧠
From pioneering vision-restoring treatments to cutting-edge CAR-T innovations, this week’s roundup of Gene and Cell Therapy breakthroughs delivers the latest on regulatory wins, funding milestones, and clinical progress shaping the future of precision medicine.
🌍 In this episode:
👁️ LUXTURNA expands globally for RPE65-related retinal dystrophies
💊 ENCELTO names Orsini as exclusive U.S. distributor
👶 Hubble Therapeutics raises $7.3M for rare pediatric blindness gene therapy
🧫 Mesoblast launches Ryoncil® for pediatric GvHD across the U.S.
🧮 Vertex halts VX-264, accelerates Zimislecel toward 2026 global launch
❤️ CardiAMP therapy shows long-term survival gains in heart failure
🧠 Voyager’s tau-targeting gene therapies advance toward IND
🌱 Public funding powers nonprofit gene therapy for CTNNB1 syndrome
⚙️ Cellares earns FDA AMT designation for its Cell Shuttle
🧪 ALA-CART CAR-T targets antigen-low tumors with long-lived T cells
📢 Stay Ahead in Gene and Cell Therapy Research!
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